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Cancer Treatment Outcomes: Are Manufacturers Focusing on the Patient?

August 1, 2013

While manufacturers have publicly stated their goal of including patient-reported outcomes (PROs) in drug development, the demands of regulators and payers are largely driving trial design and endpoint selection. The value of PRO endpoint (EP) measures is rarely criticized or rewarded in a public setting.

With better treatment options available, and people living longer after being diagnosed with cancer, more attention is being paid not only to the extension of life, but also to the quality of that extended survival. Thus, the industry is focusing more clearly on PRO inclusion in clinical trials.

GfK Bridgehead (part of GfK’s Health team) recently examined the inclusion of PRO measures as primary or secondary endpoints for the top ten cancer indications currently in Phase II/III and Phase III development. We identified 298 clinical trials for 130 drug candidates for the top ten cancer indications, in terms of number of assets in development that may launch in the next one to three years.

Non-small cell lung cancer (NSCLC) was the most active indication (24 drugs, 63 trials), and diffuse large B-cell lymphoma (DLBCL) had the fewest drugs (8). Pancreatic cancer had the fewest trials (11). We also found that 56 of the drugs are currently marketed in other indications while being concurrently developed for these new indications. The remaining drugs were unapproved, in either Phase II/III or Phase III development, or in Preregistration.

Of the 298 clinical trials for the top ten cancer indications, 42% (126) of the clinical trials collected PROs, with 55 individual PRO measures identified. The collection of PRO measures varied dramatically by cancer indication; chronic lymphocytic leukemia (CLL) took the lead, with 77% of trials collecting PROs. By contrast, multiple myeloma had the lowest proportion, with only 22% of trials collecting PROs.

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Drug manufacturers have largely not met the challenge of including PROs in oncology trials, with 58% of late-phase trials absent of such measures. In addition, many trials continue to use non-cancer or non-symptom-specific PRO measures for cancer indications. Manufacturers should consider including PROs as an opportunity to differentiate from potential competitors, especially in the case of a crowded pipeline market. Also, in some cases, payers may accept PRO endpoints as surrogate data, in absence of increased survival.

While some benefits of PRO inclusion have been identified, more research is needed to better understand the impact of PRO inclusion and selection in Phase II/III and Phase III cancer trials on clinical and commercial outcomes.

GfK presented this analysis at the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) 2013 Congress; you can download the poster presentation at this link.

Marija Ringwelski is Senior Analyst of GfK Bridgehead, which is part of GfK’s global Health team. She can be reached at

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